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Article

Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores

2022-10-27

Abstract excerpt

<title>Abstract</title> <p>Background Generalized pairwise comparisons (GPC) can be used to assess the Net Benefit of new treatments for rare diseases. We show the potential of GPC through simulations based on data from a natural history study in mucopolysaccharidosis type IIIA (MPS IIIA). Methods Using data from a historical series of untreated children with MPS IIIA aged 2 to 9 years at the time of enrolment...

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Literature Corpus work
ee950097-54cf-50e6-a192-31b0631f4e87
DOI
10.21203/rs.3.rs-1832055/v1
Open publication

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Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scoresDOI 10.21203/rs.3.rs-1832055/v1
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